Defining genes and pathways that modify huntingtin CAG repeat somatic instability in vivo

IF 31.7 1区生物学 Q1 GENETICS & HEREDITY

Nature genetics Pub Date : 2025-01-22 DOI:10.1038/s41588-024-02055-4

Suphinya Sathitloetsakun, Myriam Heiman

引用次数: 0

Abstract

A novel in vivo CRISPR screening platform identifies genetic modifiers of huntingtin CAG repeat somatic instability. These modifiers include known and novel genes that are promising therapeutic targets for Huntington’s disease.

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确定基因和途径修饰亨廷顿蛋白CAG重复体内体细胞不稳定性

一种新的体内CRISPR筛选平台识别亨廷顿蛋白CAG重复体细胞不稳定性的遗传修饰因子。这些修饰因子包括已知的和新的基因，它们有望成为亨廷顿舞蹈病的治疗靶点。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Nature genetics 生物-遗传学

CiteScore

43.00

自引率

2.60%

发文量

241

审稿时长

3 months

期刊介绍： Nature Genetics publishes the very highest quality research in genetics. It encompasses genetic and functional genomic studies on human and plant traits and on other model organisms. Current emphasis is on the genetic basis for common and complex diseases and on the functional mechanism, architecture and evolution of gene networks, studied by experimental perturbation. Integrative genetic topics comprise, but are not limited to: -Genes in the pathology of human disease -Molecular analysis of simple and complex genetic traits -Cancer genetics -Agricultural genomics -Developmental genetics -Regulatory variation in gene expression -Strategies and technologies for extracting function from genomic data -Pharmacological genomics -Genome evolution